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Past AMDA Webinars
BMN-701: Potentially More Effective Treatment Option For Pompe
Title: BMN-701: Potentially more effective treatment option for Pompe
Date: Monday, December 13, 2010
Speaker:
Barbara Wuebbels, BioMarin
Jon Lebowitz, BioMarin
Diaphragm Delivery Of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
Title: DiaphragmThe main muscle used for breathing. Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene VectorA delivery system, often based on a modified virus, used to carry genetic material into cells. in Patients With Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart.
Date: Tuesday, October 26, 2010
Speaker:
Barry J Byrne, MD, PhD – University of Florida
Autophagy and its Role in Pompe Disease
Title: AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. and it’s Role in Pompe Disease
Date: Wednesday, August 4, 2010
Speakers:
Nina Raben, MD, PhD – Staff Scientist, Arthritis + Rheumatism Branch, National Institute of Arthritis + Musculoskeletal + Skin Diseases (NIAMS), National Institutes of Health, Bethesda, MD.]